Association of factor expression levels with health-related quality of life and direct medical costs for people with haemophilia B.

From PubMed via Jisc Publications RouterPublication status: ppublishGene therapy trials aim to provide a functional cure for patients with haemophilia B (HB), and treatment impact is analyzed by factor IX expression levels (FELs). We investigated the relationship of FELs with health-related quality of life (HRQoL) and costs. This was a retrospective cross-sectional analysis of the European (CHESS I-II) and US (CHESS-US) CHESS population studies. Physicians recruited consecutive patients and extracted information from the medical records; patients completed questionnaires between 2014 and 2015 (CHESS-I), 2018-2019 (CHESS-II) and 2019 (CHESS US). Patients with inhibitors were excluded. HRQoL was assessed using the EQ-5D-5L. Twelve-month haemophilia-related direct medical costs included office visits and hospitalizations based on country-level unit costs. A Tobit model was used to analyze FELs and HRQoL and generalized linear models for direct medical costs. A total of 191 men with HB completed the EQ-5D questionnaire; the mean age was 36.8 years, with a mean FEL of 10.1 IU/dL (median, 4.0). Mean EQ-5D was 0.77 (SD, 0.23). The Tobit model adjusting for age, body mass index and blood-borne viruses showed every 1% increase in FEL was associated with +0.006 points in the mean EQ-5D score (  = .003). Mean haemophilia-related direct medical costs excluding factor replacement therapy were €2,028/year (median, €919) in CHESS I-II (EU,  = 226), and $7,171/year (median,$586) in CHESS US (  = 181). Adjusted EU and US models showed every 1% increase in FEL was associated with a decrease in haemophilia-related direct medical costs of €108/year and $529/year, respectively. Direct medical costs were based on physician extraction of encounters from medical records, potentially underestimating costs of care. The voluntary nature of participation may have introduced selection biases. We observed a significant association of increases in FEL with increased HRQoL and decreased costs in Europe and the United States among men with HB and no inhibitors

Technically measured compositional physical work demands and prospective register-based sickness absence (PODESA): a study protocol

Abstract Background Various physical work demands are shown to be associated with sickness absence. However, these studies have: (a) predominantly used self-reported data on physical work demands that have been shown to be inaccurate compared with technical measurements, (b) principally focused on various physical work demands in ‘isolation’, i.e. ignoring their co-dependency – compositional nature –, and (c) mainly used register data on long-term sickness absence. The present article describes the protocol of a study with the objective of investigating the association between technically measured compositional data on physical work demands and prospective long- and short-term register-based data on sickness absence. Methods ‘The technically measured compositional Physical wOrk DEmands and prospective association with register-based Sickness Absence study (PODESA)’ comprises data from two Danish cohorts (NOMAD and DPhacto) primarily on blue-collar workers. In the PODESA cohort, data on 1108 workers were collected at baseline (between 2011 and 2014). The cohort data comprise, e.g., self-reported information on descriptives, lifestyle, workday, and health, as well as accelerometer-based measurements of physical work demands (physical activity, movements, and postures). These baseline measurements are linked with prospective register-based data on sickness absence for up to four years after baseline. The prospective association between physical work demands and sickness absence will be analysed using a Compositional Data Analysis approach. Discussion PODESA provides a unique possibility of unravelling which combinations of physical work demands are associated with prospective sickness absence. PODESA employs technically measured information on physical work demands (taking into account the compositionality of physical work demand data) and prospective sickness absence data. The findings from PODESA can be used to develop strengthened preventive interventions for sickness absence. Results are expected in 2019–2021

Determinants of adherence and consequences of the transition from adolescence to adulthood among young people with severe haemophilia (TRANSHEMO): A multicentric French national observational cross‐sectional study based on the FranceCoag registry

International audienceAbstract Introduction It is necessary to gain insights into adherence to healthcare in people with severe haemophilia (PwSH), especially during the transition from paediatric to adult care, which is an important phase in lives of young people with childhood chronic disease. This adherence can be considered as a marker of successful transition. Objectives The main objective of the quantitative phase of the TRANSHEMO project was to compare the adherence to healthcare between adolescents and young adults (YAs) with severe haemophilia. The secondary objective was to identify the determinants (facilitators and barriers) of this adherence and associations between these determinants. Methods A multicentre, observational, cross‐sectional study was conducted in 2017–2019 on PwSH aged between 14 and 17 years (adolescents) or between 20 and 29 years (YAs), included in the FranceCoag registry and having completed the questionnaires. The adherence to healthcare (treatment regimens and clinical follow‐up) was compared between adolescents and YAs using the chi‐squared test. The determinants of this adherence were analysed by structural equation modelling. Results There were 277 participants, 107 adolescents, and 170 YAs. The rate of adolescents adhering to healthcare was 82.2%, while the rate of YAs was 61.2% ( p < .001). The barriers to the adherence to healthcare were being YA, having repeated at least one school grade and presenting mental health concerns. Conclusion Adolescents had better adherence to healthcare than YAs. According to the determinants enlightened in this project, targeted supportive strategies and adapted therapeutic education programs can be developed for young PwSH to facilitate their adherence to healthcare

Determinants of adherence and consequences of the transition from adolescence to adulthood among young people with severe haemophilia (TRANSHEMO): study protocol for a multicentric French national observational cross-sectional study

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